Another round trip! Vinay Prasad is leaving the FDA for the second time in roughly a year. His tenure brought high-profile rejections that surprised rare disease developers, raising questions about whether the FDA's messaging on flexibility matched its actions. My take: the

ARK's Big Ideas 2026 video is LIVE 🎬Jam-packed with ARK's research on the technologies reshaping the global economy. And I’m in it! I may be coming down with a cold when this was taped but don’t mind that 😊 I walk through the Multiomics section (starting ~1:04:00) — 5

The FDA just made one of the most significant shifts in its evidentiary standards in decades—and it feels like a lot of people have slept on the news! The FDA will now default to ONE pivotal trial for drug approval, not two. The science of evaluating whether a drug works has

🚦 Green light, red light — where is genetic medicine headed? Big picture: Gene editing is moving closer to first in-vivo approvals, while early-stage innovation suggests the future toolkit could enable gene-scale edits, broadly expanding the set of genetic diseases that may

Only ~2% of the human genome codes for proteins. The other 98%? Biological “dark matter.” DeepMind’s new AlphaGenome AI could help decode how non coding DNA influences gene regulation and disease, much like AlphaFold transformed protein structure prediction from amino acid

See our Big Ideas 2026 here: https://t.co/vEz7C86Wn0

How large is the potential economic value of extending healthy human life? Delaying age-related disease and functional decline could significantly expand healthy lifespans. Measured in quality-adjusted life years (QALYs), the potential gains suggest an opportunity far larger than

You know your chronological age—but how old are you biologically, and how well can we measure it and assess mortality risk? Progress in medicine has meaningfully extended lifespans by reducing early deaths from infectious and chronic diseases. As a result, mortality is now

What happens when curative therapies scale from rare disease to common disease? While gene-editing cures are initially being developed in rare diseases, the underlying technologies have the potential to scale into far more common conditions. As gene-editing approaches advance,

If you could choose—would you prefer a one-time treatment or lifelong chronic therapy? Innovations in gene-editing medicine could enable one-time treatments for certain rare diseases. While these therapies are likely to carry higher upfront prices than chronic treatments, they

And here’s a short snippet from "The Brainstorm" episode on why gene editing is at an inflection point 👇

Gene editing is at an inflection point. In-vivo CRISPR could move single-dose, functional cures beyond rare diseases to common killers like cardiovascular disease—the world’s leading cause of death. Hear me discuss the science and implications on this week’s episode of The

Gene-editing is moving from rare diseases to some of the world’s biggest killers. With in-vivo, LNP-delivered CRISPR, a one-time gene edit could potentially lower lifelong cardiovascular risk and transform treatment for millions who don’t adequately respond to current

Baby KJ need not be a one-off success story. For families who have felt left behind as their children faced similarly devastating diagnoses, the FDA’s new plausible mechanism pathway offers a reason for real hope. As described in the FDA’s NEJM paper, the pathway would allow

While there may be bumps along the way, gene-editing innovation marches on—and regulators are evolving to keep pace. The FDA is signaling a major shift: building a faster pathway for personalized gene-editing therapies by grouping related disorders into combined trials. This

It’s amazing how far we’ve come in sequencing the human genome. Last week, PacBio cut the cost of long-read sequencing by ~40% - now down to <$300 per genome at scale. Hard to believe the first human genome in 2003 cost ~$2.7BN! These breakthroughs will keep accelerating

Wow, exciting work out last week on a new gene editing system called bridge editing, which could expand the range of diseases addressable by genetic medicine. Where CRISPR fixes “typos,” bridge editing may be able to rewrite an entire chapter of DNA—up to nearly 1 million base

Eli Lilly’s new TuneLab is an interesting new paradigm for AI-powered drug discovery: federated learning lets small biotechs train on Lilly’s $1B+ AI models without exposing proprietary data. The potential result? Stronger shared models, faster & cheaper drug discovery and better

Cardiovascular disease has been the #1 killer of Americans for years. Statins became blockbusters treating it despite adherence capping commercial success. Gene editing could go further—permanently lowering risk and replacing decades of pills with ONE treatment. Your thoughts?